Patients

The global, randomized, double-blind, placebo-controlled Phase 3 RINGSIDE trial (ClinicalTrials.gov Identifier: NCT04871282) evaluated the efficacy and safety of varegacestat in patients with progressing desmoid tumors. A total of 156 patients were randomized to receive varegacestat 1.2 mg daily or placebo until disease progression or death, representing the largest randomized study in this population. The primary endpoint of the trial was progression-free survival as assessed by blinded independent central review. Statistically controlled secondary endpoints were confirmed ORR using RECIST v1.1 and change in tumor volume at week 24, both determined by blinded independent central review, as well as change in pain intensity at week 12 as determined using a patient-reported outcome instrument. Additional secondary endpoints included duration of response, best reduction in tumor volume, patient-reported outcomes, and safety and tolerability. RINGSIDE includes an open-label extension phase, which is ongoing.

The Phase 1 clinical trial of IM-1021 is ongoing, with the first patient dosed in February 2025. The Phase 1 trial is an open-label, multicenter dose-escalation and expansion study that is expected to include participants with advanced B-cell lymphomas and advanced solid tumors.

• Desmoid tumors, also known as aggressive fibromatosis or desmoid-type fibromatosis, are aggressive, non-metastatic soft tissue tumors that are prone to recurrence.

• Depending on where in the body they occur, desmoid tumors can cause debilitating pain, deformity and, in some cases, life threatening organ damage.

• Approximately 1,000-1,650 people are diagnosed with desmoid tumors each year in the United States, and there are approximately 10,000-11,000 actively managed patients.

• Cases typically occur in people between the ages of 15 and 60 and are most common in early adulthood, with a peak around 30 years of age They are more common in women than men.

• The pain and physical limitations associated with desmoid tumors lead to high clinical burdens and impaired quality of life.

Expanded access, also called compassionate use, is a potential pathway for patients with an immediate life-threatening or serious condition to gain early access to an investigational product for treatment outside of clinical trials when no comparable or satisfactory alternative therapy option is available.

Immunome is a clinical-stage oncology company committed to developing first- and best-in-class targeted therapies designed to improve outcomes for cancer patients. We are focused on conducting clinical trials that evaluate the safety and efficacy of our investigational therapies and generate data to support their regulatory approval and broad availability to patients who may benefit from them. Immunome is not making our investigational therapies available via expanded access at this time. We believe that participation in our clinical trials will be the most appropriate way to access our investigational therapies prior to their approval. More information about our clinical trials can be found on ClinicalTrials.gov.

Immunome will continue to assess our expanded access policy and provide updates to this website. Please contact us with any questions at info@immunome.com.